July 24, 2020July 24, 2020
FAQs About a Kidney Transplant for Fabry Disease
FAQs
Fabry disease is a rare genetic disease characterized by the buildup of a fatty molecule — globotriaosylceramide Gb3 or GL-3 ... Read more
July 22, 2020July 14, 2020
Fabry Disease: A Road Less Traveled
Columns, Fabry: A Road Less Traveled – a Column by Jerry Walter
For my introductory Fabry Disease News column, I’d like to share a little about me and my journey with ... Read more
July 21, 2020July 21, 2020
Certain Fabry Patients May Switch from ERT to Galafold, Real-life Study Suggests
News
Switching from enzyme replacement therapy (ERT) to Galafold (migalastat) is a valid and safe therapeutic option for Fabry disease patients with ... Read more
July 17, 2020July 15, 2020
A Look at Fabry Through My Daughter’s Eyes
Columns, The Fabry Files - a Column by Eunice White
My 12-year-old daughter was diagnosed with Fabry disease in spring 2019. Because she’s relatively new to having Fabry, I ... Read more
July 14, 2020July 14, 2020
Alternative Therapies Needed for Patients Who Respond Poorly to ERT, Case Report Says
News
A case study of two cousins with Fabry disease who started producing high levels of neutralizing antibodies after enzyme ... Read more
July 14, 2020July 14, 2020
The Fabry Registry
Health Insights
If you have Fabry disease, you should consider joining the Fabry Registry. Among other benefits, the information you can ... Read more
July 9, 2020July 9, 2020
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
News, syndicated
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the ... Read more
July 7, 2020July 7, 2020
Comprehensive New Urine Test May Help Doctors Monitor Fabry Disease Progression
News
A newly developed urine test that measures the levels of different proteins may help doctors to more quickly and ... Read more
June 30, 2020June 30, 2020
Single Dose of AMT-190 Effective in Mice and Primate Studies of Fabry
News
A single administration of AMT-190, uniQure’s investigational gene therapy for Fabry disease, significantly increases the activity of alpha-galactosidase A ... Read more
June 26, 2020June 26, 2020
Women With Fabry Disease
Health Insights
Fabry disease is a rare genetic disorder that is caused by mutations in the GLA gene. The body uses the instructions ... Read more
June 23, 2020June 23, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
cystic fibrosis, News, syndicated
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during ... Read more
June 19, 2020June 19, 2020
FAQs About Kidney Health and Dialysis in Fabry Disease
FAQs
Fabry disease is a rare disease characterized by the buildup of a fatty molecule (globotriaosylceramide Gb3 or GL-3) inside ... Read more

July 24, 2020July 24, 2020

FAQs About a Kidney Transplant for Fabry Disease

FAQs

Fabry disease is a rare genetic disease characterized by the buildup of a fatty molecule — globotriaosylceramide Gb3 or GL-3 — inside cells, interfering with their function. The accumulation of ... Read more

July 22, 2020July 14, 2020

Fabry Disease: A Road Less Traveled

Columns, Fabry: A Road Less Traveled – a Column by Jerry Walter

For my introductory Fabry Disease News column, I’d like to share a little about me and my journey with Fabry disease. Just over 40 years ago, in 1979, Fabry ... Read more

July 21, 2020July 21, 2020

Certain Fabry Patients May Switch from ERT to Galafold, Real-life Study Suggests

News

Switching from enzyme replacement therapy (ERT) to Galafold (migalastat) is a valid and safe therapeutic option for Fabry disease patients with Galafold-amenable mutations, according to a real-life study from Italy. Notably, ... Read more

July 17, 2020July 15, 2020

A Look at Fabry Through My Daughter’s Eyes

Columns, The Fabry Files - a Column by Eunice White

My 12-year-old daughter was diagnosed with Fabry disease in spring 2019. Because she’s relatively new to having Fabry, I thought it’d be interesting to collect her thoughts on life ... Read more

July 14, 2020July 14, 2020

Alternative Therapies Needed for Patients Who Respond Poorly to ERT, Case Report Says

News

A case study of two cousins with Fabry disease who started producing high levels of neutralizing antibodies after enzyme replacement therapy (ERT) highlight the need for alternative therapies. The ... Read more

July 14, 2020July 14, 2020

The Fabry Registry

Health Insights

If you have Fabry disease, you should consider joining the Fabry Registry. Among other benefits, the information you can provide may help scientists develop new treatments for the genetic ... Read more

July 9, 2020July 9, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease ... Read more

July 7, 2020July 7, 2020

Comprehensive New Urine Test May Help Doctors Monitor Fabry Disease Progression

News

A newly developed urine test that measures the levels of different proteins may help doctors to more quickly and accurately monitor the progression of Fabry disease, a study finds. ... Read more

June 30, 2020June 30, 2020

Single Dose of AMT-190 Effective in Mice and Primate Studies of Fabry

News

A single administration of AMT-190, uniQure’s investigational gene therapy for Fabry disease, significantly increases the activity of alpha-galactosidase A — the faulty enzyme in Fabry patients — in mice ... Read more

June 26, 2020June 26, 2020

Women With Fabry Disease

Health Insights

Fabry disease is a rare genetic disorder that is caused by mutations in the GLA gene. The body uses the instructions that this gene provides to make an enzyme (alpha-galactosidase A) ... Read more